Evaluation of Treatment Outcomes of High‐Risk Gestational Trophoblastic Neoplasms (GTNs)

ABSTRACT This study was performed to assess the efficacy, safety, and prognostic factors of treatment protocols in patients with high‐risk gestational trophoblastic neoplasia (GTN). Data of 64 high‐risk GTN patients treated at our clinic between January 1993 and December 2020 were retrospectively analyzed. Patients were classified according to the FIGO staging and World Health Organization (WHO) scoring system, and demographic characteristics, treatment protocols (etoposide, methotrexate, actinomycin‐D, cyclophosphamide, vincristine [EMACO]; methotrexate, actinomycin‐D, cyclophosphamide [MAC]; etoposide, methotrexate, actinomycin‐D, etoposide, cisplatin [EMAEP]), complications, and survival outcomes were evaluated. The presence of metastatic disease, beta‐human chorionic gonadotropin (β‐hCG) levels, and treatment responses were analyzed in detail. The median age of the patients was 31.5 years (19–57), and 65.6% had metastatic disease. The most commonly used chemotherapy protocol was EMACO (75% of patients). Primary chemotherapy failure was seen in 42.1%, and the median number of cycles given until β‐hCG reset was 3 (1–9). Complete remission was achieved in 90.6% of the patients, whereas 9.4% died. The most common complications were neutropenia (24.3%), elevated liver function tests (17.6%), and anemia (17.6%). The presence of brain metastasis and a high WHO score (11–15) were significantly associated with mortality ( p < 0.05). The EMACO protocol is a good option for the treatment of high‐risk GTN; however, the prognosis is worse, particularly in patients with brain metastases and a high WHO score. Improving the diagnosis and choosing the appropriate treatment protocol are crucial for the management of this disease. Summary It provides real‐world data on treatment outcomes from a substantial cohort of high‐risk GTN patients treated at a single institution. It offers detailed analysis of chemotherapy‐related complications across different regimens, information that is often underreported in the literature. It identifies specific patient subgroups who may benefit from alternative treatment approaches, potentially guiding more tailored management strategies. It contributes to the ongoing discussion about the limitations of current FIGO scoring and staging systems.